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Muscular Dystrophy - Pipeline Review, H1 2016

Published: May, 2016 | Pages: 135 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Muscular Dystrophy - Pipeline Review, H1 2016


Global Markets Direct's, 'Muscular Dystrophy - Pipeline Review, H1 2016', provides an overview of the Muscular Dystrophy pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Muscular Dystrophy, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Muscular Dystrophy and features dormant and discontinued projects. 

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.


- The report provides a snapshot of the global therapeutic landscape of Muscular Dystrophy
- The report reviews pipeline therapeutics for Muscular Dystrophy by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Muscular Dystrophy therapeutics and enlists all their major and minor projects
- The report assesses Muscular Dystrophy therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Muscular Dystrophy

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Muscular Dystrophy
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Muscular Dystrophy pipeline depth and focus of Indication therapeutics
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and Scope
- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline
 Table of Contents
Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Muscular Dystrophy Overview 8 Therapeutics Development 9 Pipeline Products for Muscular Dystrophy - Overview 9 Pipeline Products for Muscular Dystrophy - Comparative Analysis 10 Muscular Dystrophy - Therapeutics under Development by Companies 11 Muscular Dystrophy - Therapeutics under Investigation by Universities/Institutes 13 Muscular Dystrophy - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Muscular Dystrophy - Products under Development by Companies 17 Muscular Dystrophy - Products under Investigation by Universities/Institutes 20 Muscular Dystrophy - Companies Involved in Therapeutics Development 21 Acceleron Pharma, Inc. 21 AMO Pharma Limited 22 Asahi Kasei Pharma Corp. 23 aTyr Pharma, Inc. 24 Benitec Biopharma Limited 25 Bioblast Pharma Ltd. 26 BioMarin Pharmaceutical Inc. 27 Biophytis SAS 28 Evotec AG 29 F. Hoffmann-La Roche Ltd. 30 Fate Therapeutics, Inc. 31 Genethon 32 Ionis Pharmaceuticals, Inc. 33 Marina Biotech, Inc. 34 Novogen Limited 35 Prothelia, Inc. 36 SanBio, Inc. 37 Santhera Pharmaceuticals Holding AG 38 Sarepta Therapeutics, Inc. 39 Selecta Biosciences, Inc. 40 Takeda Pharmaceutical Company Limited 41 WAVE Life Sciences Ltd. 42 Muscular Dystrophy - Therapeutics Assessment 43 Assessment by Monotherapy Products 43 Assessment by Target 44 Assessment by Mechanism of Action 46 Assessment by Route of Administration 48 Assessment by Molecule Type 50 Drug Profiles 52 ACE-083 - Drug Profile 52 AMO-02 - Drug Profile 54 Antisense Oligonucleotide to Activate Dystrophin for Muscular Dystrophy - Drug Profile 55 Antisense Oligonucleotide to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 56 Antisense RNAi Oligonucleotides for Myotonic Dystrophy - Drug Profile 57 ATYR-1940 - Drug Profile 58 BIO-101 - Drug Profile 60 BIO-103 - Drug Profile 61 Drugs for Merosin-Deficient Congenital Muscular Dystrophy Type 1A - Drug Profile 62 elcatonin - Drug Profile 63 Gene Therapy for Muscular Dystrophy and Liver Diseases - Drug Profile 64 Gene Therapy to Activate Dysferlin for Duchenne and Limb Girdle Muscular Dystrophies - Drug Profile 65 Gene Therapy to Activate Dysferlin for Dysferlinopathies - Drug Profile 66 Gene Therapy to Activate Dystrophin for Muscular Dystrophy - Drug Profile 67 IONISDMPK-2.5Rx - Drug Profile 68 IUCT-169 - Drug Profile 70 IUCT-290 - Drug Profile 71 IUCT-309 - Drug Profile 72 ketoprofen - Drug Profile 73 LR-08 - Drug Profile 74 Oligonucleotide 1 to Target Dystrophia Myotonica Protein Kinase for Myotonic Dystrophy - Drug Profile 75 Oligonucleotides to Inhibit DM1 Protein Kinase for Myotonic Dystrophy - Drug Profile 76 omigapil - Drug Profile 77 Pabparna - Drug Profile 79 PRO-135 - Drug Profile 80 PRT-01 - Drug Profile 81 Recombinant Protein to Activate Utrophin for Muscular Dystrophies - Drug Profile 83 RNAi Gene Therapy to Inhibit Myotilin for LGMD - Drug Profile 84 RP-33 - Drug Profile 85 SB-308 - Drug Profile 86 Small Molecule to Target CUG RNA for Myotonic Dystrophy 1 - Drug Profile 87 Small Molecule to Target RNA for Myotonic Dystrophy - Drug Profile 88 Small Molecules for Dysferlinopathies - Drug Profile 89 Small Molecules for Facioscapulohumeral Muscular Dystrophy - Drug Profile 90 Small Molecules for Myotonic Dystrophy - Drug Profile 91 Small Molecules for Myotonic Dystrophy Type 1 - Drug Profile 92 Small Molecules to Activate SMCHD1 for Facioscapulohumeral Dystrophy - Drug Profile 94 Small Molecules to Inhibit MBNL1 for Myotonic Dystrophy Type I - Drug Profile 95 Small Molecules to Target RNA for Myotonic Dystrophy - Drug Profile 96 SRT-149 - Drug Profile 97 SRT-152 - Drug Profile 98 Stem Cell Therapy for Muscular Dystrophy - Drug Profile 99 Stem Cell Therapy for Musculoskeletal Disorders - Drug Profile 100 Stryka-232 - Drug Profile 101 Stryka-234 - Drug Profile 102 Stryka-425 - Drug Profile 103 Stryka-533 - Drug Profile 104 Stryka-978 - Drug Profile 105 trehalose - Drug Profile 106 VAL-0411 - Drug Profile 107 VAL-1205 - Drug Profile 108 Muscular Dystrophy - Recent Pipeline Updates 109 Muscular Dystrophy - Dormant Projects 117 Muscular Dystrophy - Discontinued Products 119 Muscular Dystrophy - Product Development Milestones 120 Featured News & Press Releases 120 Appendix 130 Methodology 130 Coverage 130 Secondary Research 130 Primary Research 130 Expert Panel Validation 130 Contact Us 130 Disclaimer 131
List of Tables
Number of Products under Development for Muscular Dystrophy, H1 2016 13 Number of Products under Development for Muscular Dystrophy - Comparative Analysis, H1 2016 14 Number of Products under Development by Companies, H1 2016 15 Number of Products under Development by Companies, H1 2016 (Contd..1) 16 Number of Products under Investigation by Universities/Institutes, H1 2016 17 Comparative Analysis by Late Stage Development, H1 2016 18 Comparative Analysis by Clinical Stage Development, H1 2016 19 Comparative Analysis by Early Stage Development, H1 2016 20 Products under Development by Companies, H1 2016 21 Products under Development by Companies, H1 2016 (Contd..1) 22 Products under Development by Companies, H1 2016 (Contd..2) 23 Products under Investigation by Universities/Institutes, H1 2016 24 Muscular Dystrophy - Pipeline by Acceleron Pharma, Inc., H1 2016 25 Muscular Dystrophy - Pipeline by AMO Pharma Limited, H1 2016 26 Muscular Dystrophy - Pipeline by Asahi Kasei Pharma Corp., H1 2016 27 Muscular Dystrophy - Pipeline by aTyr Pharma, Inc., H1 2016 28 Muscular Dystrophy - Pipeline by Benitec Biopharma Limited, H1 2016 29 Muscular Dystrophy - Pipeline by Bioblast Pharma Ltd., H1 2016 30 Muscular Dystrophy - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 31 Muscular Dystrophy - Pipeline by Biophytis SAS, H1 2016 32 Muscular Dystrophy - Pipeline by Evotec AG, H1 2016 33 Muscular Dystrophy - Pipeline by F. Hoffmann-La Roche Ltd., H1 2016 34 Muscular Dystrophy - Pipeline by Fate Therapeutics, Inc., H1 2016 35 Muscular Dystrophy - Pipeline by Genethon, H1 2016 36 Muscular Dystrophy - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 37 Muscular Dystrophy - Pipeline by Marina Biotech, Inc., H1 2016 38 Muscular Dystrophy - Pipeline by Novogen Limited, H1 2016 39 Muscular Dystrophy - Pipeline by Prothelia, Inc., H1 2016 40 Muscular Dystrophy - Pipeline by SanBio, Inc., H1 2016 41 Muscular Dystrophy - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2016 42 Muscular Dystrophy - Pipeline by Sarepta Therapeutics, Inc., H1 2016 43 Muscular Dystrophy - Pipeline by Selecta Biosciences, Inc., H1 2016 44 Muscular Dystrophy - Pipeline by Takeda Pharmaceutical Company Limited, H1 2016 45 Muscular Dystrophy - Pipeline by WAVE Life Sciences Ltd., H1 2016 46 Assessment by Monotherapy Products, H1 2016 47 Number of Products by Stage and Target, H1 2016 49 Number of Products by Stage and Mechanism of Action, H1 2016 51 Number of Products by Stage and Route of Administration, H1 2016 53 Number of Products by Stage and Molecule Type, H1 2016 55 Muscular Dystrophy Therapeutics - Recent Pipeline Updates, H1 2016 113 Muscular Dystrophy - Dormant Projects, H1 2016 121 Muscular Dystrophy - Dormant Projects (Contd..1), H1 2016 122 Muscular Dystrophy - Discontinued Products, H1 2016 123

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